Errant Gene Therapeutics, LLC (EGT), a pioneering boutique drug development firm specializing in Rare Diseases, announced the transfer of its clinical grade lentiviral vector, TNS 9.55.3, to Memorial Sloan Kettering Cancer Centre (MSKCC). TNS 9.55.3 developed by EGT pursuant to an exclusive license agreement with Sloan Kettering Institute (SKI), will be used for MSKCC's upcoming beta Thalassemia human clinical trial.

The trial protocol provides for the “in vitro” treatment of beta-thalassemia patients, offering the prospect for a cure for a chronic condition which would otherwise subject the patient to early death without incurring life-long regular blood transfusions and chelation therapy.

A new definitive agreement between EGT and SKI provides for MSKCC to lead the clinical trials and arrange for drug development partnerships for the treatment of beta-Thalassemia and Sickle Cell Disease, two of the worlds most prevalent and devastating hereditary blood conditions.

EGT continues its decade's long commitment to cure beta-Thalassemia, also called Cooley's Anaemia, through dedicated outreach to patient and research communities around the world. Pat Girondi, EGT founder and CEO, stated, “the pioneering research of SKI's Sadelain team is positioned to deliver the safest and most effective cure for the many millions of patients whose hopes and prayers we have honoured with our total dedication.”

EGT received protocol approval for the upcoming trials by unanimous vote of the Recombinant DNA Advisory Committee of the NIH in 2007, and was granted orphan drug designation by both the FDA and the EMEA. The clinical trial is expected to commence in the fourth quarter of 2011.

Sam Salman, EGT's president said that with “MSKCC and SKI taking the lead in the clinical and developmental activities for TNS 9.55.3, EGT will be able to further progress its pre-clinical programs for its novel drug cancer therapies targeting chronic hormone refractory cancers.”

Errant Gene Therapeutics (EGT) is a privately held biopharmaceutical company and a pioneer in the emerging field of epigenetics focused on the treatment of deadly hormone  refractory cancers using its patented portfolio of small molecule Histone Deacetylases inhibitors (HDACi).